Gene Therapy Net - News, Conferences, Vectors, Literature, Clinical Trials and morehttp://www.genetherapynet.com/
Mon, 21 Jan 2019 20:10:50 +0000Joomla! - Open Source Content Managementen-gbWelcome to Gene Therapy Nethttp://www.genetherapynet.com/215-frontpage/2-welcome-to-gene-therapy-net.html
http://www.genetherapynet.com/215-frontpage/2-welcome-to-gene-therapy-net.htmlGene Therapy Net is the web resource for patients and professionals interested in gene therapy. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international regulations and guidelines associated with clinical gene therapy trials.

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]]>info@genetherapynet.com (Administrator)FeaturedFrontpageFri, 31 Dec 1999 23:00:00 +0000Gene Therapy Drug Approval to Skyrocket by 2025http://www.genetherapynet.com/gene-therapy-news/614-gene-therapy-drug-approval-to-skyrocket-by-2025.html
http://www.genetherapynet.com/gene-therapy-news/614-gene-therapy-drug-approval-to-skyrocket-by-2025.htmlPosted on: 16 January 2019, source: FDAThe FDA expects to approve 10 to 20 cell and gene therapy products a year within the next six years, and is hiring 50 more clinical reviewers to make it happen, the agency announced Jan. 15: "The FDA is witnessing a surge of cell and gene therapy products entering early development, evidenced by a large upswing in the number of investigational new drug (IND) applications. Based on this activity, we anticipate that the number of product approvals for cell and gene therapies will grow in the coming years, reflecting significant scientific advancement and the clinical promise of these new innovations."

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{{Google468x60Tekst}}]]>info@genetherapynet.com (Administrator)FeaturedSpecial News ItemsWed, 16 Jan 2019 04:00:00 +0000 Gene therapy for Crigler-Najjar syndrome - First patient treated in the European clinical trialhttp://www.genetherapynet.com/gene-therapy-news/613-gene-therapy-for-crigler-najjar-syndrome-first-patient-treated-in-the-european-clinical-trial.html
http://www.genetherapynet.com/gene-therapy-news/613-gene-therapy-for-crigler-najjar-syndrome-first-patient-treated-in-the-european-clinical-trial.htmlPosted on: 25 December 2018, source: EurekalertsFollowing patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.

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]]>info@genetherapynet.com (Administrator)FeaturedSpecial News ItemsTue, 25 Dec 2018 04:00:00 +0000Chinese Researchers Claim to Have Genetically Engineered the First HIV-Immune Babieshttp://www.genetherapynet.com/gene-therapy-news/600-chinese-researchers-claim-to-have-genetically-engineered-the-first-hiv-immune-babies.html
http://www.genetherapynet.com/gene-therapy-news/600-chinese-researchers-claim-to-have-genetically-engineered-the-first-hiv-immune-babies.htmlPosted on: 28 November 2018, source: fortune.comResearchers in China (Clinical project 'Safety and validity evaluation of HIV immune gene CCR5 gene editing in human embryos') used genetic engineering tools (CRISPR) to create twins theoretically immune to HIV, smallpox, and cholera, MIT Technology Review reported. The medical breakthrough is controversial, as many worry about eugenics and designer babies for the wealthy. The twins, named Lulu and Nana, according to lead scientist He Jiankui of Shenzhen in a YouTube video, were the result of in vitro fertilization (IVF). A few weeks old, they appear to be healthy. When they were a single cell, genetic surgery using a popular tool, CRISPR, “removed the doorway through which HIV enters to infect people.”

Watch the presentation of He Jiankui and discussion during Second International Summit on Human Genome Editing in Hongkong, Wednesday November 28th, 2018.

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]]>info@genetherapynet.com (Administrator)FeaturedSpecial News ItemsTue, 27 Nov 2018 04:00:00 +0000Spark Therapeutics' Luxturna Gets EU Approval for Vision Losshttp://www.genetherapynet.com/gene-therapy-news/599-spark-therapeutics-luxturna-gets-eu-approval-for-vision-loss.html
http://www.genetherapynet.com/gene-therapy-news/599-spark-therapeutics-luxturna-gets-eu-approval-for-vision-loss.htmlPosted on: 26 November 2018, source: NasdaqSpark Therapeutics, Inc. announced that the European Commission has approved the marketing authorization application ("MAA") for its gene therapy, Luxturna (voretigene neparvovec). The MAA sought approval for the therapy in adult as well as pediatric patients as a one-time treatment for vision loss due to inherited retinal dystrophy caused by a genetic mutation in both copies of the RPE65-gene and who have enough viable retinal cells. The RPE65 mutations, a progressive disease, can lead to total blindness.

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]]>info@genetherapynet.com (Administrator)FeaturedSpecial News ItemsMon, 26 Nov 2018 04:00:00 +0000Gene therapy drugs step toward approval in Japanhttp://www.genetherapynet.com/gene-therapy-news/597-gene-therapy-drugs-step-toward-approval-in-japan.html
http://www.genetherapynet.com/gene-therapy-news/597-gene-therapy-drugs-step-toward-approval-in-japan.htmlPosted on: 14 November 2018, source: Nikkei AsiaNovartis has applied with Japanese authorities to have a gene therapy drug approved, a move that could give Japan at least two such drugs in the coming year, Nikkei has learned. The Switzerland-based pharmaceutical hopes to receive the all-clear in Japan for a neurological disorder treatment sometime next year. It has also applied for approval for the same drug in the U.S. and Europe. In the U.S., the drug is said to be prohibitively expensive -- as much as $4 million to $5 million per patient. The drug treats spinal muscular atrophy, and Novartis expects to be able to market the treatment as early as next year.

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]]>info@genetherapynet.com (Administrator)FeaturedSpecial News ItemsWed, 14 Nov 2018 04:00:00 +0000Infectivity of adeno-associated virus serotypes in mouse testishttp://www.genetherapynet.com/gene-therapy-news/596-infectivity-of-adeno-associated-virus-serotypes-in-mouse-testis.html
http://www.genetherapynet.com/gene-therapy-news/596-infectivity-of-adeno-associated-virus-serotypes-in-mouse-testis.htmlPosted on: 7 November 2018, source: BMC BiotechnologyIn a recent study, researchers from the Institute for Stem Cell Biology and Regenerative Medicine (InStem), Bengaluru, National Centre for Biological Sciences (NCBS), Bengaluru, Christian Medical College, Vellore and the Indian Institute of Technology, Kanpur, have described how a type of virus, called adeno-associated virus, behaves when injected into the mouse testis. The findings of the study, supported by the Department of Biotechnology (DBT) and InStem were published in the journal BMC Biotechnology.

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]]>info@genetherapynet.com (Administrator)FeaturedSpecial News ItemsWed, 07 Nov 2018 04:00:00 +0000FDA Lifts Clinical Hold; Green-Lights Vertex and CRISPR’s Sickle Cell Gene Therapy Trialhttp://www.genetherapynet.com/gene-therapy-news/595-fda-lifts-clinical-hold-green-lights-vertex-and-crispr%E2%80%99s-sickle-cell-gene-therapy-trial.html
http://www.genetherapynet.com/gene-therapy-news/595-fda-lifts-clinical-hold-green-lights-vertex-and-crispr%E2%80%99s-sickle-cell-gene-therapy-trial.htmlPosted on: 14 October 2018, source: BioSpaceThe U.S. Food and Drug Administration (FDA) has lifted a clinical hold and accepted an Investigational New Drug Application (IND) for an experimental sickle cell disease treatment being co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. In May the FDA placed a hold on the trial and IND for CTX001, an investigational gene editing treatment, citing concerns over questions that had not been addressed in the IND. The two companies initially submitted the IND in April in support of a planned Phase I/II trial.